The FDA seeks a cure for stem cell quackery

David Weissberg of Long Island Orthopedic Solutions: Stem cell therapy is a science still finding its legs.

March 18, 2016

By GREGORY ZELLER //

With federal regulators looming and a charged FDA hearing set for later this year, the nation’s stem cell community is tense, particularly clinicians incorporating stem cell therapies in private practice.

Scientifically, stem cells are on the rise. Therapies cultivating stem cells from a patient’s own body are increasingly common treatments for autism, various cancers, joint injuries and a host of other ailments; nutritionists push supplements infused with stem cells, goji berry and green tea.

Internationally, the scope grows wider: Some form of stem-cell research or practice has occurred in virtually every modernized nation, applied to conditions ranging from myocardial infarction to deafness, from diabetes to HIV, even infertility and baldness.

This despite raging domestic controversy about the use of stem cells. While some of the hoopla is based on misconceptions – recalling the embryonic stem cell debates of decades past – the controversy has left patients and practitioners on edge and has set the stage for the Food and Drug Administration hearing.

Stem cells are “undifferentiated biological cells,” or primordial cells that have not yet assumed their functional characteristics – they don’t know what they want to be when they grow up. They will eventually differentiate into specialized cells, and they can multiply through mitosis.

Common in multicellular organisms, stem cells come in two varieties in mammals: embryonic stem cells and adult stem cells. Most U.S. practitioners extract adult stem cells from patients, purify them through nonchemical means and reinject them into the same host, but “stem cell” still carries a stigma related to the use of embryonic stem cells, which has generated significant philosophical, moral and religious objections.

Years ago, according to orthopedic surgeon Dr. David Weissberg, whose Long Island Orthopedic Solutions has offered stem cell therapies at its Huntington and West Islip offices for about a year, “the public heard ‘stem cells,’ and everyone thought you were slaughtering fetuses and encouraging abortions.”

The strong emotions engendered by such stark associations are hard to shake. But fetal cells and related ethical issues “have absolutely nothing to do with the cells we get today,” Weissberg noted, and neither does the “dilemma” facing current practitioners.

That has more to do with a lack of hard scientific data proving the efficacy of stem cell therapies – beyond reported patient responses, which are generally good – and questions about the legitimacy of the FDA approval processes.

For instance: Researchers wishing to engage a stem cell study must submit a proposal to the FDA for an Institutional Review Board, an independent ethics committee that formally approves, monitors and reviews biomedical research in humans or animals.

Weissberg, a graduate of Stony Brook University and Brooklyn’s Downstate Medical School, has not gone through the IRB process, because he still has “questions about the legitimacy” of the administration’s stem cell approvals and “I don’t want to be the subject of an FDA investigation.”

Instead, the surgeon limits his practice’s use of stem cells to techniques well within established legal parameters – and he’s double-careful about making claims regarding stem cell efficacy, even though several patients report decreased pain after stem cell therapies on degenerative joints.

“I won’t make a claim that stem cells actually regrow cartilage, because the scientific data is still being developed,” Weissberg said. “But with arthritis, when you lose cartilage, the joint space narrows. And I have seen X-rays of joints that have been treated with stem cells, and the joint space has widened.”

“There are not a lot of studies that prove the efficiency,” Gargiulo told Innovate LI. “But clinically, patients do show significant improvement. Do we improve the cartilage? I’m not sure. But patients definitely experience a significant reduction in pain.”

Adding to the national controversy are a handful of cases in which patients experienced traumatic effects, or worse, from stem cell-related treatments. There was a 2012 headline-maker involving a Florida cardiologist who had his license suspended after a patient died following a stem cell procedure. That same year, Scientific American cited a case in which a 60-something Los Angeles resident developed painful bone fragments in her eyelid following a stem-cell facelift.

And in 2014, New Scientist reported on a paralyzed American citizen who underwent stem cell treatments in Brazil and grew mucous-secreting nasal tissue on her spine, ultimately requiring surgery to remove a 3-centimeter growth and bits of bone.

For every one of those cases, there are literally tens of thousands of FDA-approved treatments in which, as Gargiulo noted, “the worst thing you can say is they have no effect.”

Like Weissberg, Gargiulo – who studied medicine in Uruguay and later trained in anesthesia and pain-management at the Westchester Medical Center – has never had a patient report any adverse effects from stem cell therapies, which AgeFocus has been offering for about four years.

He also agrees with Weissberg that stem cell therapies are a natural extension of therapies involving platelet-rich plasma, another science that needed to find its legs before gaining mainstream acceptance.

Gargiulo likened PRP to the fertilizer to the stem cell’s seed, and referenced the evolution of PRP therapies as a possible arc for stem cell proliferation.

“The PRP we could obtain 15 years ago was not as good as what we can obtain right now,” he noted. “We can obtain much purer PRP today.”

While the FDA hammers out more concrete stem cell policies, both practitioners said they would continue to perform same-day procedures in which stem cells are removed from bone marrow or fat cells (a much richer supply, Weissberg noted), purified in a centrifuge and reinjected into the target area, such as a balky knee.

What they can’t do, Weissberg noted, is manipulate the cells chemically. Bone marrow contains a relatively low percentage of stem cells, and methods for obtaining the most stem cells from fat – as much a 1,000 times what can be harvested from marrow – require the use of collagenase or other enzymes.

Using what are called “culture expansion” techniques, doctors can also produce an endless supply of stem cells that “can be grown and then frozen and stored for use by any individual for the indefinite future, which could prove to be a life saver,” Weissberg said.

In some countries, that science is in regular practice. But the FDA has issued “very strict guidelines” regarding cultural expansion of stem cells, according to Weissberg, who’s careful to steer clear.

While he does remove stem cells from fat and puts them in a centrifuge to remove excess liquid, the FDA requires “a high level of certification, which the average practitioner won’t have,” to introduce those enzymes and create “pallets of pure stem cells.”

“When you put that into a joint, you’re giving a significant dose of stem cells with a high probability of a good outcome,” Weissberg said. “But the FDA absolutely prohibits that, unless you’re a high-end, certified FDA lab.”

Some doctors obtain stem cells from foreign sources, but with no way to verify the stem cell count, or if the cells are even still alive, Weissberg avoids that, too. However, he said he “knows of doctors who are skirting the issue” and suggested “there are many around the country doing it.”

Hence the FDA crackdown, which includes the recent issuance of “Homologous Use of Human Cells, Tissues, and Cellular and Tissue-Based Products – Draft Guidance for Industry and FDA Staff,” and the planned hearing in Maryland.

Despite pressure from a concerned public, mainstream physicians challenging unproven stem cell therapies and even Big Pharma – “There’s no money in stem cells for them and eventually, their pharmaceuticals won’t be needed,” Gargiulo noted – Weissberg doesn’t expect much to change at the hearing.

Gargiulo, meanwhile, predicts “a little more regulation, and I’m not against that.” If nothing else, he added, a public vetting could finally dismiss embryonic concerns and theories about stem cell “quackery.”

“That’s not a very responsible attitude,” the doctor said. “There is not a single study that proves there is harm in using your body’s own stem cells, and there hasn’t been a single case where they can prove something went wrong from injecting an individual’s own stem cells into that person.

“The responsible thing is to organize some multi-center studies,” Gargiulo added. “Then we can follow-up and examine the results.”